Hello KIF1A.ORG Community,

Want to know what’s more valuable than money? Data. In rare diseases, data are inherently difficult to collect and analyze. But we rely on data to inform everything from creating better care guidelines to discovering treatments and cures. We can’t treat what we don’t know. Which symptoms do patients experience, and why do some patients experience certain symptoms while others do not? What is the rate of disease progression? Which outcomes should we measure to evaluate potential therapeutics? Data is the currency that purchases those answers.

Thanks to the engagement of patients and families affected by KIF1A Associated Neurological Disorder, we gain a better understanding of these questions every day. Since KIF1A.ORG was established in 2017, our family community has worked closely with Dr. Wendy Chung and her team at Columbia University to gather invaluable patient data. One of the reasons why we chose the Chung Lab to steward our precious data is Dr. Chung’s commitment to data sharing and open science.

It’s this commitment to patients that led KIF1A Associated Neurological Disorder to become one of the first rare diseases included in an FDA-funded initiative created to accelerate therapeutic development!

According to the U.S. FDA, the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is “an integrated database and analytics hub designed to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection. The aim is to receive and protect data from a variety of sources that can inform rare disease characterization, clinical trial design and other critical questions in rare disease drug development.”

Source: https://c-path.org/programs/rdca-dap/

Key stakeholders in the drug development process will be able to access KAND data through RDCA-DAP®, including governmental regulatory agencies, pharmaceutical companies, biotech companies, and academia. That means even more scientists and institutions dedicated to developing treatments and cures for rare diseases will have access to validated, standardized KAND data.   

A list of diseases in RDCA-DAP® – Source: RDCA-DAP® Quarterly Newsletter | October 11, 2022

We thank the entire team at the Chung Lab for working every day to improve our understanding of KAND, and for so readily sharing data and insights with the scientific and medical community. If you are a KAND patient or family member, you can take an active role in research from the comfort of your own home or by participating in clinical studies at Columbia University. Visit our Participate in Research page to learn more.

We may be rare, but we have a fleet of scientists in our KIF1A Research Network dedicated to making treatments and cures a reality for this generation of KAND patients. Our participation in RDCA-DAP® will offer KIF1A Research Network members even more tools and recruit new members to join our relentless mission of changing the fate of KAND.

Relentlessly,

Kathryn Atchley
President, KIF1A.ORG
kathryn@kif1a.org

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